Hopes that Amryt’s Filsuvez (AP101) might show efficacy in epidermolysis bullosa had dimmed after the readout from the pivotal Ease trial was delayed, in part to increase enrolment. So today’s announcement of a hit on the primary endpoint came as a surprise, sending shares in the London and Nasdaq-listed company up 40%, to a record high. Significantly more patients in the Filsuvez arm had complete closure of the target wound within 45 days (p=0.013) than those on placebo, Amryt said. Secondary measures failed to hit statistical significance, although “favourable differences” were observed. Puzzlingly, considering the hit on the primary, the key secondary of time to target wound closure over 90 days was not met. Still, executives claimed on a call that Filsuvez “undoubtedly had an effect on acceleration of wound healing”. Full presentation of the data will be keenly awaited to see how close these missed endpoints came, while the numbers behind the primary endpoint also remain important. Still, given the intractable nature of this devastating skin disease, and lack of options, Filsuvez seems to have a good chance of getting to market, something Amryt hopes to see happen before the end of 2021.
The Ease trial: awaiting further details. | |
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Primary endpoint | Outcome |
Proportion of patients with complete closure of the target wound within 45 ± 7 days of treatment | Hit (p=0.013) |
Key secondary endpoint | |
Time to wound closure up to 90 ± 7 days of treatment | Missed |
Other secondary endpoints | |
Incidence of first complete wound closure of EB target wound at different time points | Missed |
Change from baseline in EB target wound size | Missed |
Change in total body wound burden over time | Missed |
Change in percentages of TBSA affected by EB partial thickness wounds | Missed |
Patient reported outcomes | |
Change from baseline in background and procedural pain after wound dressing change | |
Change from baseline in itching before wound dressing change | |
Response to treatment | |
Change from baseline in sleep quality | |
Number of days missed from school or work | |
Safety endpoints | |
Incidence, severity, and relatedness of AEs | |
Local tolerability | |
Laboratory findings | |
Incidence and severity of wound infections | |
Source: Study design and rationale. |